New Data Presented At ECFS Conference Highlight Potential for Denufosol to Provide Benefit to Adolescent Cystic Fibrosis Patients and Patients on Minimal Pharmacotherapies
DURHAM, N.C.--(BUSINESS WIRE)--Jun 17, 2010 - Inspire Pharmaceuticals, Inc. (NASDAQ: ISPH) announced today that data is being presented on denufosol tetrasodium, an investigational therapy for cystic fibrosis (CF), during an oral presentation and poster presentations at the 33rd Annual European Cystic Fibrosis Society (ECFS) Conference June 16 - 19, 2010 in Valencia, Spain. The data from Inspire's first Phase 3 clinical trial with denufosol, TIGER-1, suggest that denufosol, an inhaled ion channel regulator, may ameliorate the accelerated loss of lung function in CF patients during adolescence and has the potential to provide significant benefit in lung function for those patients on minimal pharmacotherapies.
“During adolescence, CF patients are especially vulnerable to an accelerated rate of decline in lung function and there is a consequent need for therapies that delay or reduce lung function loss in this population,” stated Richard B. Moss, M.D., Professor Emeritus of Pediatric Pulmonary Medicine at Stanford University School of Medicine and Co-Director of the Children's Health Research Program at Stanford University Medical Center. “The significant lung function improvements observed in the subgroup of adolescent patients in the TIGER-1 clinical trial suggest the potential of denufosol as an early disease-stage intervention therapy that may reduce the loss of lung function during this critical time in a CF patient's lung disease progression.”
The oral presentation, “Denufosol Improved Lung Function in Adolescent CF Patients” (R.B. Moss, A. Schaberg, C. Deans, W. Tian, L. Smiley, N. Herje, T. Durham, F.J. Accurso), is being presented during the session, “Workshop 5: Current Clinical Trials,” on Thursday, June 17 from 3:30 - 3:45 p.m. local time (9:30 - 9:45 a.m. ET). The presentation highlights subgroup data from the pre-specified analysis of adolescent CF patients aged 12-18 years old (n=123) from Inspire's first Phase 3 clinical trial with denufosol, TIGER-1. In this trial, denufosol significantly improved lung function versus placebo in adolescents during the 24-week double-blind portion of TIGER-1 and continued improvement in lung function was observed with open-label treatment. The change from baseline in FEV1 (Forced Expiratory Volume in One Second) for the adolescent patients treated with denufosol at the week-24 endpoint was 112 mL compared to -10 mL for placebo (p=0.013). In comparison, in the intent-to-treat (ITT) population in TIGER-1 (n=352), the change from baseline in FEV1 at the 24-week endpoint was 48 mL for denufosol compared to 3 mL for placebo (p=0.047). The rate of decline in percent predicted FEV1 for adolescent patients receiving placebo from week 0 to week 24 during the TIGER-1 trial was significantly different from zero (p <0.001; corresponding to a decline over the trial period) while the rate of decline in percent predicted FEV1 for adolescent patients receiving denufosol was not significantly different from zero (p=0.134). The change from baseline for FEF25%-75% (Forced Expiratory Flow), a measure of small airways function, for the adolescent patients treated with denufosol at the week-24 endpoint was 115 mL/sec. compared to -112 mL/sec. for placebo (p=0.036).
The Company is also presenting three additional posters at the ECFS Conference. The poster presentation, “Potential of Denufosol as an Early Intervention in CF Lung Disease: Efficacy in Patients with Minimal Pharmacotherapy in a U.S. Phase 3 Clinical Trial” (F.J. Accurso, W. Tian, A. Schaberg, T. Navratil, M.S. Howenstine, T.G. Liou), examines the efficacy of denufosol in patients taking zero to two classes of concomitant medication at baseline for CF lung disease in the TIGER-1 trial (n=71). These patients had baseline FEV1 scores of approximately 92% predicted normal, comparable to the ITT population in the trial. In this post-hoc analysis, denufosol demonstrated benefit compared to placebo in pulmonary function tests and quality of life respiratory score in those patients with minimal pharmacotherapy. For the week-24 endpoint of FEV1 in liters, the treatment effect in the subgroup was 100 mL (p=0.059) and the treatment effect in percent predicted FEV1 was 6.4% (p=0.011). These results highlight the potential of denufosol to be used early in the CF lung disease process.
Inspire is presenting a poster presentation entitled, “Aerosol and Pharmacokinetic Properties of Denufosol Support its Use for Early Intervention in CF Lung Disease” (T. Navratil, A. Schaberg, F. Johnson, T. Durham, C.L. Ren, F. Ratjen, R.B. Moss. F.J. Accurso), which summarizes in vitro data that suggest that denufosol, as a result of its aerosol and chemical characteristics, has the potential to reach the small airways of the lungs, where CF lung disease begins. This was supported by a post-hoc analysis of the TIGER-1 trial data, which showed a significant improvement relative to placebo in FEF25%-75% in a subgroup of patients (n=329) with baseline FEV1 ‰¤110% predicted normal (p=0.025). In the TIGER-1 trial, following inhalation, there was little to no evidence of systemic exposure to denufosol and no evidence of accumulation as measured by plasma levels.
Inspire is presenting a poster presentation entitled, “Analysis of Polypharmacy in Patients with Mild Cystic Fibrosis Lung Disease Assigned to Placebo in Phase 3 Clinical Trial of Denufosol” (T. Durham, T. Navratil, A. Schaberg, C. Deans, L. Smiley, N. Herje, F. J. Accurso). As an assessment of the magnitude of concomitant medication use independent of treatment effects with denufosol, a post-hoc analysis of patients assigned to placebo during the TIGER-1 clinical trial (n=175) was conducted. This analysis demonstrated that more than 71% of patients were taking five or more concomitant medications for treatment of CF lung disease, not including those medications used to treat gastrointestinal or other aspects of CF disease. In this analysis, the extent of concomitant medication use was as high in patients with better lung function (FEV1 ‰¥90% predicted) as it was in those with lower lung function (FEV1 ‰¥75% to <90% predicted). The development of disease modifying therapies that address the underlying cause of CF lung disease may reduce the need for extensive polypharmacy.
These presentations will be available on Inspire's website, www.inspirepharm.com, following the conference.
About Denufosol Tetrasodium
Denufosol is a first-in-class ion channel regulator targeted as an early intervention treatment that potentially corrects the ion transport defect in patients regardless of CF genotype. Denufosol is designed to enhance airway hydration and mucociliary clearance by increasing chloride secretion, inhibiting sodium absorption and increasing ciliary beat frequency. These integrated pharmacological actions and the potential to reach the small airways are key to maintaining lung function and potentially delaying the progression of lung disease.
Inspire is currently conducting TIGER-2, its second Phase 3 clinical trial with denufosol, and expects top-line results from this trial in the first quarter of 2011. Assuming the results of TIGER-2 are positive, that Inspire subsequently files a New Drug Application (NDA) for denufosol with the FDA and that the FDA approves such NDA, Inspire is targeting a potential U.S. commercial launch for denufosol in the 2012 timeframe.
Inspire is a biopharmaceutical company focused on researching, developing and commercializing prescription pharmaceutical products for ophthalmic and pulmonary diseases. Inspire's goal is to build and commercialize a sustainable portfolio of innovative new products based on its technical, scientific and commercial expertise. The most advanced compounds in Inspire's clinical pipeline are denufosol tetrasodium for cystic fibrosis and PROLACRIA™ (diquafosol tetrasodium ophthalmic solution) 2% for dry eye, which are both in Phase 3 development, and AZASITE® (azithromycin ophthalmic solution) 1% for blepharitis, which is in Phase 2 development. Inspire receives revenues related to the promotion of AZASITE for bacterial conjunctivitis, the co-promotion of ELESTAT® (epinastine HCl ophthalmic solution) 0.05% for allergic conjunctivitis and royalties based on net sales of RESTASIS® (cyclosporine ophthalmic emulsion) 0.05% for dry eye. For more information, visit www.inspirepharm.com.
The forward-looking statements in this news release relating to management's expectations and beliefs are based on preliminary information and management assumptions. Specifically, no assurance can be made with respect to: the ability of denufosol tetrasodium to ameliorate the accelerated loss of lung function in CF patients during adolescence and to provide significant benefit in lung function for those patients on minimal pharmacotherapies; the potential of denufosol as an early disease-stage intervention therapy that may reduce the loss of lung function at any time in a CF patient's lung disease progression; the potential of denufosol to be used early in the CF lung disease process; the potential ability of denufosol as a result of aerosol and chemical characteristics to reach the small airways of the lung; the development of disease modifying therapies that address the underlying cause of CF lung disease may reduce the need for extensive polypharmacy; the timing and availability of top-line results from TIGER-2; the success of the TIGER-2 clinical trial; the timing of an NDA for denufosol, if any; FDA approval of any such NDA; the timing of any potential commercial launch for denufosol in 2012 or otherwise; and whether Inspire will successfully be able to build and commercialize a sustainable portfolio of innovative new products based on its technical, scientific and commercial expertise. Such forward-looking statements are subject to a wide range of risks and uncertainties that could cause results to differ in material respects, including those relating to product development, revenue, expense and earnings expectations, the seasonality of ELESTAT, intellectual property rights, competitive products, results and timing of clinical trials, success of marketing efforts, the need for additional research and testing, delays in manufacturing, funding, and the timing and content of decisions made by regulatory authorities, including the U.S. Food and Drug Administration. Further information regarding factors that could affect Inspire's results is included in Inspire's filings with the SEC. Inspire undertakes no obligation to publicly release the results of any revisions to these forward-looking statements that may be made to reflect events or circumstances after the date hereof.
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Posted: June 2010